Trial designs using real‐world data: The changing landscape of the regulatory approval process

National Impact of 13-Valent Pneumococcal Conjugate Vaccine on Ambulatory Care Visits for Otitis Media in Children under 5 Years in the United States

(2019) Xiaofeng Zhou et al.   INTERNATIONAL JOURNAL OF PEDIATRIC OTORHINOLARYNGOLOGY

Blinatumomab for minimal residual disease in adults with B-cell precursor acute lymphoblastic leukemia

(2018) Nicola Gökbuget et al.   BLOOD

Innovation at the Intersection of Clinical Trials and Real-World Data Science to Advance Patient Care

(2018) Brandon Swift et al.   CTS-Clinical and Translational Science

Real-world evidence research based on big data

(2018) Benedikt E. Maissenhaelter et al.   Onkologe

Advancing a Framework for Regulatory Use of Real-World Evidence

(2018) Nancy A. Dreyer   Therapeutic Innovation & Regulatory Science

FDA Approval Summary: Axicabtagene Ciloleucel for Relapsed or Refractory Large B-cell Lymphoma

(2018) Najat Bouchkouj et al.   CLINICAL CANCER RESEARCH

Predictive model algorithms identifying early and advanced stage ER+/HER2− breast cancer in claims data

(2018) Daniel C. Beachler et al.   PHARMACOEPIDEMIOLOGY AND DRUG SAFETY

Complete Hematologic and Molecular Response in Adult Patients With Relapsed/Refractory Philadelphia Chromosome–Positive B-Precursor Acute Lymphoblastic Leukemia Following Treatment With Blinatumomab: Results From a Phase II, Single-Arm, Multicenter Study

(2017) Giovanni Martinelli et al.   JOURNAL OF CLINICAL ONCOLOGY

Untapped Potential of Observational Research to Inform Clinical Decision Making: American Society of Clinical Oncology Research Statement

(2017) Kala Visvanathan et al.   JOURNAL OF CLINICAL ONCOLOGY

Axicabtagene Ciloleucel CAR T-Cell Therapy in Refractory Large B-Cell Lymphoma

(2017) Sattva S. Neelapu et al.   NEW ENGLAND JOURNAL OF MEDICINE

What Is Real-World Data? A Review of Definitions Based on Literature and Stakeholder Interviews

(2017) Amr Makady et al.   VALUE IN HEALTH

Good Practices for Real‐World Data Studies of Treatment and/or Comparative Effectiveness: Recommendations from the Joint ISPOR‐ISPE Special Task Force on Real‐World Evidence in Health Care Decision Making

(2017) Marc L. Berger et al.   VALUE IN HEALTH

Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

(2016) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

A pragmatic analysis comparing once-monthly paliperidone palmitate versus daily oral antipsychotic treatment in patients with schizophrenia

(2016) Larry Alphs et al.   SCHIZOPHRENIA RESEARCH

Blinatumomab vs historical standard therapy of adult relapsed/refractory acute lymphoblastic leukemia

(2016) N Gökbuget et al.   Blood Cancer Journal

GetReal: from efficacy in clinical trials to relative effectiveness in the real world

(2016) Matthias Egger et al.   Research Synthesis Methods

Real-World Outcomes of Paliperidone Palmitate Compared to Daily Oral Antipsychotic Therapy in Schizophrenia

(2015) Larry Alphs et al.   JOURNAL OF CLINICAL PSYCHIATRY

Big Data Analytics in Healthcare

(2015) Ashwin Belle et al.   Biomed Research International

Randomised controlled trials and population-based observational research: partners in the evolution of medical evidence

(2014) C M Booth et al.   BRITISH JOURNAL OF CANCER

Design and Rationale of the Paliperidone Palmitate Research in Demonstrating Effectiveness (PRIDE) Study

(2014) Larry Alphs et al.   JOURNAL OF CLINICAL PSYCHIATRY

Creative trial design in RA: optimizing patient outcomes

(2013) Maya H. Buch et al.   Nature Reviews Rheumatology

Is the Large Simple Trial Design Used for Comparative, Post-Approval Safety Research?

(2011) Robert F. Reynolds et al.   DRUG SAFETY

Evaluating the Evidence

(2008) P. Michael Ho et al.   CIRCULATION